CC-4047 in Treating Patients With Myelofibrosis

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00669578
First received: April 29, 2008
Last updated: June 20, 2014
Last verified: June 2014
Results First Received: November 7, 2012  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Chronic Myeloproliferative Disorders
Secondary Myelofibrosis
Intervention: Drug: CC-4047

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This study opened June 2008 and accrued 12 Phase I participants and 65 Phase II participants before being closed in January 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants were accrued to the Phase I portion of this study in each of the 2.5, 3.0, and 3.5 mg dose cohorts. Dose Limiting Toxicities (DLT) were observed at the 3.5 mg level, and the 3 mg level was confirmed as the maximum tolerated dose. However, efficacy was not improved at higher doses and the most effective dose level was deemed 0.5 mg/day.

Reporting Groups
  Description
Phase I Participants were accrued to the Phase I portion of this study in each of the 2.5, 3.0, and 3.5 mg dose cohorts to determine the Maximum Tolerated Dose (MTD). Dose limiting Toxicities (DLT) were observed at the 3.5 mg level, and the 3 mg level was confirmed as the maximum tolerated dose. For this study a DLT was defined as a grade 4 hematologic toxicity, a grade 3 or higher febrile neutropenia, or a grade 3 or higher non-hematologic toxicity.
Phase II All patients enrolled to this phase II arm started treatment at 0.5mg/day with CC-4047 every day of each 28 day cycle.

Participant Flow:   Overall Study
    Phase I     Phase II  
STARTED     12     65  
COMPLETED     12     65  
NOT COMPLETED     0     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Phase II All patients enrolled to this Phase II arm started treatment at 0.5mg/day with CC-4047 every day of each 28 day cycle.
Phase I Participants were accrued to the Phase I portion of this study in each of the 2.5, 3.0, and 3.5 mg dose cohorts to determine the Maximum Tolerated Dose (MTD). Dose limiting Toxicities (DLT) were observed at the 3.5 mg level, and the 3 mg level was confirmed as the maximum tolerated dose. For this study a DLT was defined as a grade 4 hematologic toxicity, a grade 3 or higher febrile neutropenia, or a grade 3 or higher non-hematologic toxicity.
Total Total of all reporting groups

Baseline Measures
    Phase II     Phase I     Total  
Number of Participants  
[units: participants]
  65     12     77  
Age  
[units: years]
Median ( Full Range )
  68  
  ( 42 to 87 )  
  66  
  ( 51 to 83 )  
  67  
  ( 42 to 87 )  
Gender  
[units: participants]
     
Female     18     7     25  
Male     47     5     52  
Region of Enrollment  
[units: participants]
     
United States     65     12     77  



  Outcome Measures
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1.  Primary:   Determine the Maximum Tolerated Dose of CC-4047   [ Time Frame: The first 28-day cycle of treatment. ]

2.  Primary:   Best Overall Response Over the First 6 Cycles of Treatment   [ Time Frame: Every cycle of treatment for 6 cycles. Each cycle is 28 days. ]

3.  Secondary:   Number of Participants With Treatment Related Adverse Events.   [ Time Frame: During treatment and every 6 months until 3 years from registration or progression. ]

4.  Secondary:   Duration of Response Time   [ Time Frame: Time from response to disease progression, intolerance of study drug, or death. ]

5.  Secondary:   Time to Response   [ Time Frame: Time from registration to the first date of response within twelve 28-day cycles of treatment. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Ayalew Tefferi, M.D.
Organization: Mayo Clinic
e-mail: tefferi.ayalew@mayo.edu


No publications provided


Responsible Party: Mayo Clinic
ClinicalTrials.gov Identifier: NCT00669578     History of Changes
Other Study ID Numbers: MC078B, P30CA015083, MC078B, NCI-2009-01331, 07-005317, PO-MMM-PI-0007
Study First Received: April 29, 2008
Results First Received: November 7, 2012
Last Updated: June 20, 2014
Health Authority: United States: Food and Drug Administration